期刊
VISION RESEARCH
卷 111, 期 -, 页码 124-133出版社
PERGAMON-ELSEVIER SCIENCE LTD
DOI: 10.1016/j.visres.2014.07.013
关键词
Retina; Gene therapy; AAV; Lentiviral vector; LCA; Clinical trial
资金
- NIH [1DP1OD008267-01]
- Curing Kids Fund
- Foundation for Retina Research
- Foundation Fighting Blindness
- Research to Prevent Blindness
The maturity in our understanding of the genetics and the pathogenesis of disease in degenerative retinal disorders has intersected in past years with a novel treatment paradigm in which a genetic intervention may lead to sustained therapeutic benefit, and in some cases even restoration of vision. Here, we review this prospect of retinal gene therapy, discuss the enabling technologies that have led to first-in-human demonstrations of efficacy and safety, and the road that led to this exciting point in time. (C) 2014 Elsevier Ltd. All rights reserved.
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